Spinal cord injury (SCI) damages ascending and descending axons that are unable to regenerate and re-establish functional connections with their targets. In addition to paralysis and loss of sensory function below the level of the lesion, SCI may also lead to chronic pain, spasticity, respiratory impairment, loss of bowel or bladder control, and sexual dysfunction. Currently, there is no clinically effective therapy available. In addition, therapeutic strategies are needed to treat cancer.
Gene therapy involves the intracellular delivery of a vector containing genetic material capable of expressing a therapeutic gene. Delivery of therapeutics and/or nucleic acids capable of changing gene expression levels may be desirable.